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Aplastic Anemia and MDS International Foundation, Perspective from a funder, Alice Houk

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Aplastic Anemia and MDS International Foundation. Alice Houk, Senior Director of Patient and Professional Services

Preface:

We’re thrilled to share our recent interview with Alice Houk, Senior Director of Patient and Professional Services at the Aplastic Anemia and MDS International Foundation (AAMDSIF). AAMDSIF is the leading nonprofit organization dedicated to supporting people affected by aplastic anemia, myelodysplastic syndromes (MDS), paroxysmal nocturnal hemoglobinuria (PNH), and other related bone marrow failure diseases. This amazing organization provides crucial support and resources to patients and their families around the world, and plays a key role in funding and advocating for research to discover better treatments and potential cures.


In this interview, we delve into the mission of AAMDSIF, explore the funding opportunities available for researchers, and get some valuable advice from Alice for those interested in applying for funding. If you want to learn more about the impactful work of AAMDSIF and how they’re driving advancements in medical research and patient support, we warmly invite you to read this insightful interview.

Could you provide an overview of the mission of AAMDSIF?

The Aplastic Anemia and MDS International Foundation is the world’s leading nonprofit health organization dedicated to supporting patients and families living with aplastic anemia, myelodysplastic syndrome (MDS), paroxysmal nocturnal hemoglobinuria (PNH), and related bone marrow failure diseases. The Foundation provides answers, support, and hope to thousands of patients and their families around the world.

AAMDSIF advocates for and invests in collaborative research to accelerate the discovery of better treatments and cures for bone marrow failure disorders. 

Tell us more about your funding opportunities available for researchers from AAMDSIF?

The Aplastic Anemia and MDS International Foundation, Inc. (AAMDSIF) is pleased to offer research grants to investigators dedicated to studying bone marrow failure and advancing the understanding, prevention and treatment of  bone marrow disorders in both pediatric and adult populations. Since 1989, AAMDSIF has provided financial support for research that leads to new insights into the causes of  bone marrow failure diseases and to the development of new therapeutic approaches.  The 2024  program will accept applications in the following types of research:

  •       Basic research
  •       Translational research
  •       Pre-clinical research (e.g. drug development)
  •       Early-stage clinical research
  •       Health services research/epidemiology

Could you kindly inform whether the AAMDSIF Research Grant Program for the year 2024 is currently open for applications? Additionally, are there specified categories for which proposals in 2024 will be considered?

In 2024, AAMDSIF will award up to four grants in the following categories:

A)    Aplastic Anemia, MDS and PNH

AAMDSIF will accept proposals addressing these bone marrow failure disorders:

  • Aplastic Anemia (focus on acquired)
  • Myelodysplastic Syndromes/Neoplasms (MDS)
  • MDS transition to Acute Myeloid Leukemia (AML)
  •  Paroxysmal Nocturnal Hemoglobinuria (PNH)
  • Inherited bone marrow failure syndromes

Funding is available to support up to two projects at $60,000 over two years ($30,000 per year) judged to have the highest scientific merit by the AAMDSIF Medical Advisory Board reviewers.  

B)    Pediatric Aplastic Anemia

AAMDSIF will accept proposals investigating how genetic determinants of pediatric bone marrow failure conditions can be applied to advance therapies.  This may include:

  • Research addressing a deeper comprehension of genetic mutations for the development of more personalized therapies, which may include bone marrow transplantation
  • ·Study of genetic mutations and relevant therapeutic interventions in aplastic anemia and other bone marrow failure diseases that can benefit pediatric patients.

One grant in the amount of $60,000 over two years ($30,000 each year) will be awarded to an eligible national or international investigator for a qualified project selected by the AAMDSIF Medical Advisory Board reviewers.  

C)  Graft-vs-Host Disease

In addition, proposals addressing the prevention or treatment of graft-vs-host disease (GVHD) related to hematopoietic stem cell transplantation for bone marrow failure disease will be accepted. One grant in the amount of $60,000 over two years ($30,000 each year) will be awarded to an eligible national or international investigator for a qualified project selected by the AAMDSIF Medical AdvisoryBoard reviewers.  

Do you accept applications from researchers globally?

Yes

Could you provide additional details regarding the two-step submission process employed by AAMDSIF for applications, Alice?

Letters of Intent (LOI) will be accepted from potential applicants.  The LOI deadline is June 30, 2024.  LOIs must be submitted via the online grant application portal, Proposal Central:  https://proposalcentral.altum.com/.

Application

Select applicants will be invited to submit full proposals in early September, based on review of the letters of intent. Applications will be due October 31, 2024, and must be submitted via the online grant portal ProposalCENTRAL, https://proposalcentral.altum.com/

Could you elaborate on the review process for Letters of Intent (LOIs), specifically detailing the individuals responsible for the review and the criteria they consider during the evaluation?

An NIH-model peer-review process is used to evaluate applications. Reviewers of LOIs and grant applications are members of the AAMDSIF Medical Advisory Board. It is the AAMDSIF policy not to disclose the names of the individuals who have reviewed a specific application or to provide reviewer comments. 

Awards are based on the scientific quality of the research plan, the relevance of the proposed research to the AAMDSIF goals, the applicant’s qualifications, and the quality of the research institution and facility where the research will be conducted. The quality and support of the research mentor will also be considered.

In addition, the following criteria will be considered by reviewers:

  • The likelihood that the goals of the proposal are attainable with the funding from AAMDSIF;
  • The project is independent research and not part of a large, multi-funded study;
  • The translational nature of the research and whether the findings can be rapidly applied to benefit patients in the near term (e.g. within 3 years)
  • The likelihood that the project will contribute toward the professional development of the researcher.

What amounts are allocated to beneficiaries of your grants, and will these amounts be comparable to previous years in 2024?

AAMDSIF grants are two-year, $60,000 grants ($30,000 per year) which is comparable to the amount of grants awarded in previous years.

What advice do you have for researchers interested to apply for your funding?

To be eligible to apply for a research grant, an applicant must hold a Doctor of Medicine, Doctor of Philosophy, Doctor of Osteopathy, Doctor of Science, or other doctoral level degree (e.g., PharmD). There are no nationality restrictions. Research may be conducted in the United States or abroad but not at a for-profit laboratory in the private sector. The project must represent independent research conducted by the applicant. Members of the AAMDSIF Medical Advisory Board are not eligible to apply for a grant and may sponsor no more than one applicant during any given round.

Preference will be given to New Investigators who propose a project that will contribute toward their professional development in bone marrow failure disease research. New Investigators are post-doctoral fellows, instructors, and those who have been assistant professors for fewer than five (5) years.

The Foundation is interested in funding research across the spectrum of basic and clinical science, but highest priority is for studies that have the potential to have an impact on prevention, diagnosis, risk-stratification, treatment, or complications from transplantation for these diseases. Examples of this type of research include but are not limited to:

  • Discovery of biomarkers predicting response or resistance to existing therapies
  • Correlative science on patient samples collected from clinical trials, to inform understanding of therapeutic mechanisms
  •  Late-stage pre-clinical research, with a goal of establishing proof-of-concept for a novel therapy
  •  Basic research that reveals new mechanisms of disease likely to benefit patients
  • Genetics of bone marrow failure conditions including both inherited and acquired mutations

Investigators should be prepared to provide a rationale for the research as related to a gap in evidence for the proposed project. Applicants should clearly explain why the proposed research will address an important question that has not been answered by previous research.

 

Acknowledgment

We would like to express our gratitude to Alice Houk, Senior Director of Patient and Professional Services at AAMDSIF, for this insightful interview. We also express our appreciation to the AAMDSIF organization for their amazing work supporting people affected by aplastic anemia, MDS, PNH and related bone marrow failure disorders.

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